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Applied Therapeutics APLT announced that the FDA has issued a Complete Response Letter (CRL) for the new drug application (NDA) seeking approval for APLT’s lead candidate, govorestat, to treat classic galactosemia. The CRL stated that the FDA has reviewed the application and cannot approve the NDA in its current form due to deficiencies in the clinical study results submitted in support of the application.
Applied Therapeutics is evaluating the FDA's feedback. It intends to promptly request a meeting to discuss the requirements for a potential NDA resubmission or appeal of the decision, and appropriate next steps. Shares of the company tanked 77.3% in the after-market hours, following the disappointing news.
APLT’s govorestat (also known as AT-007) is a novel central nervous system penetrant ARI, which is currently being developed for three rare neurological diseases. The NDA for govorestat for the galactosemia indication was accepted by the FDA in late February under its Priority Review pathway.
Year to date, shares of Applied Therapeutics have skyrocketed 155.8% against the industry’s 7.8% decline.
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Per APLT, clinical study results submitted in support of the govorestat NDA showed rapid and sustained reductions in galactitol upon treatment with the candidate, leading to meaningful clinical benefits for pediatric patients and a favorable safety profile. In the phase III ACTION-Galactosemia Kids study in children with galactosemia aged 2-17 years, govorestat improved daily activities, behavior, cognition, fine motor skills and tremor. It also significantly reduced plasma galactitol levels in adults and children with galactosemia. Additional studies provided strong efficacy and safety data across 185 classic galactosemia patients over three years.
Per Applied Therapeutics, approximately 3,300 patients in the United States with 80-100 new births per year and approximately 4,400 patients in the EU with 120 new births per year suffer from galactosemia, a rare genetic metabolic disease.
A regulatory filing for govorestat to treat classic galactosemia is also currently under review in the EU. The company expects a decision by the EU regulatory body early in the first quarter of 2025.
Other Programs in APLT’s Clinical-Stage Pipeline
Apart from thegalactosemia indication, Applied Therapeutics is currently evaluating govorestat for two other indications, namely sorbitol dehydrogenase (SORD) deficiency and phosphomannomutase 2 deficiency (PMM2-CDG).
The company is gearing up to submit an NDA with the FDA for govorestat to treat the SORD indication early in the first quarter of 2025. Applied Therapeutics clarified that the review and potential approval of govorestat for treating SORD is independent of the ongoing review of govorestat for classic galactosemia.